Gaucher Disease

BETHESDA, Md., April 30, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced an oral poster presentation at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, being held May 7th-10th in New York City, NY. Details of the oral and poster presentation are as follows: Presentation Title: GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson’s Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+ Presenter: Joanne Taylor, Ph.D., Senior Vice President of Research, Gain Therapeutics Session: Oral Poster Presentation – Guided Poster TourSession Date: Saturday, May 10, 2025Session Time: 8:00-9:00 a.m. EST About GT-02287Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced ER stress, lysosomal and mitochondrial pathology, aggregated α-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting. Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with a >50% increase in glucocerebrosidase (GCase) activity among those receiving GT-02287 at clinically relevant doses. GT-02287 is currently being evaluated in a Phase 1b clinical trial for the treatment of Parkinson’s disease with or without a GBA1 mutation. The primary endpoint of the trial, which is currently enrolling participants across 7 sites in Australia, is to evaluate the safety and tolerability of GT-02287 after 3 months of dosing in people with Parkinson’s disease. Gain’s lead program in Parkinson’s disease has been awarded funding support early in its development from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency. About Gain Therapeutics, Inc.Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher’s disease, dementia with Lewy bodies, and Alzheimer’s disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors. Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. Forward-Looking StatementsThis release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; the timing of any submissions to the FDA or other regulatory bodies and agencies; and the potential therapeutic and clinical benefits of the Company’s product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s Form 10-K for the year ended December 31, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@gaintherapeutics.com LifeSci Advisors LLCChuck PadalaManaging Directorchuck@lifesciadvisors.com Media:Russo Partners LLC Nic Johnson and Elio Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(760) 846-9256

NEW YORK, NY, USA I April 25, 2025 I Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced that the first patient has been administered both the first protein dose and the 177 Lu-DOTA imaging dose in its Phase 1 clinical trial evaluating the Company’s Self-Assembly and Disassembly (“SADA”) Pre-targeted Radioimmunotherapy (“PRIT”) platform for the treatment of patients with relapsed or refractory non-Hodgkin Lymphoma (r/r NHL). This Phase 1 trial (Trial 1201) is a dose-escalation, open-label, single-arm, multi-center trial investigating the safety and tolerability of the CD38-SADA: 177 Lu-DOTA Drug Complex. Trial 1201 is designed to investigate the pre-targeted delivery of the CD38-SADA protein that binds with high affinity to lymphoma cells, followed by the administration of a radioactive 177 Lu-DOTA payload to selectively target the tumor-bound CD38-SADA molecules while minimizing radiation to normal tissues. Part A of the trial is CD38-SADA dose escalation with fixed 177 Lu-DOTA payload doses to explore the optimal CD38-SADA protein dose and interval between the SADA protein administration and the payload. The primary endpoints of Part A include tumor imaging and occurrence of dose limiting toxicities in the dose limiting toxicities (DLT) evaluation period. “We are excited to announce the dosing of the first patient in Trial 1201 in patients with relapsed or refractory non-Hodgkin Lymphoma, our second clinical program evaluating the SADA PRIT platform and our first in hematological malignancies,” said Norman LaFrance, M.D., Chief Development and Medical Officer. “Relapsed and refractory NHL presents significant challenges for patients facing limited treatment options and a more aggressive disease course. We believe that our innovative approach to pre-targeted radioimmunotherapy has the potential to significantly improve outcomes in this high-risk population.” In addition to CD38-SADA, the modular design of the SADA PRIT platform has facilitated the clinical development of other bispecific fusion proteins, including GD2-SADA, now in clinical development for the treatment of GD2-expressing tumors. Researchers at MSK, including Dr. Nai-Kong Cheung, developed the SADA technology for radioimmunotherapy, which is exclusively licensed by MSK to Y-mAbs. Dr. Cheung has intellectual property rights and interests in the technology, and as a result of this licensing arrangement, MSK has institutional financial interests in the technology. About Y-mAbs Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, radioimmunotherapy and antibody-based therapeutic cancer products. The Company’s technologies include its investigational Self-Assembly DisAssembly (“SADA”) Pretargeted Radioimmunotherapy Platform (“PRIT”), and bispecific antibodies generated using the Y-BiClone platform. The Company’s broad and advanced product pipeline includes the anti-GD2 therapy DANYELZA® (naxitamab-gqgk), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow after a partial response, minor response, or stable disease to prior therapy. About CD38-SADA PRIT CD38-SADA is a bispecific fusion protein that tightly binds to the CD38 glycoprotein and to 177 Lu-tetraxetan ( 177 Lu -DOTA), a “caged” radionuclide. In the first step of pre-targeted radioimmunotherapy, non-radiolabeled CD38-SADA tetramers are infused and bind to CD38-expressing lymphoma cells, and unbound CD38-SADA protein disassembles into low molecular weight monomers that are removed by the kidney. The second infusion delivers the “radioactive payload,” which binds directly to CD38-SADA on tumor cells for localized irradiation. CD38-SADA PRIT with 177 Lu-DOTA has demonstrated robust anti-tumor efficacy in preclinical studies and is currently being investigated in adults with relapsed, progressive, or refractory NHL (CD38-expressing B-cell, T-cell, and natural killer cell lymphomas) after at least 2 prior lines of therapy ( NCT05994157 ). SOURCE: Y-mAbs

NEW YORK, April 25, 2025 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced that the first patient has been administered both the first protein dose and the 177Lu-DOTA imaging dose in its Phase 1 clinical trial evaluating the Company’s Self-Assembly and Disassembly (“SADA”) Pre-targeted Radioimmunotherapy (“PRIT”) platform for the treatment of patients with relapsed or refractory non-Hodgkin Lymphoma (r/r NHL). This Phase 1 trial (Trial 1201) is a dose-escalation, open-label, single-arm, multi-center trial investigating the safety and tolerability of the CD38-SADA: 177Lu-DOTA Drug Complex. Trial 1201 is designed to investigate the pre-targeted delivery of the CD38-SADA protein that binds with high affinity to lymphoma cells, followed by the administration of a radioactive 177Lu-DOTA payload to selectively target the tumor-bound CD38-SADA molecules while minimizing radiation to normal tissues. Part A of the trial is CD38-SADA dose escalation with fixed 177Lu-DOTA payload doses to explore the optimal CD38-SADA protein dose and interval between the SADA protein administration and the payload. The primary endpoints of Part A include tumor imaging and occurrence of dose limiting toxicities in the dose limiting toxicities (DLT) evaluation period. “We are excited to announce the dosing of the first patient in Trial 1201 in patients with relapsed or refractory non-Hodgkin Lymphoma, our second clinical program evaluating the SADA PRIT platform and our first in hematological malignancies,” said Norman LaFrance, M.D., Chief Development and Medical Officer. “Relapsed and refractory NHL presents significant challenges for patients facing limited treatment options and a more aggressive disease course. We believe that our innovative approach to pre-targeted radioimmunotherapy has the potential to significantly improve outcomes in this high-risk population.” In addition to CD38-SADA, the modular design of the SADA PRIT platform has facilitated the clinical development of other bispecific fusion proteins, including GD2-SADA, now in clinical development for the treatment of GD2-expressing tumors. Researchers at MSK, including Dr. Nai-Kong Cheung, developed the SADA technology for radioimmunotherapy, which is exclusively licensed by MSK to Y-mAbs. Dr. Cheung has intellectual property rights and interests in the technology, and as a result of this licensing arrangement, MSK has institutional financial interests in the technology. About Y-mAbs Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, radioimmunotherapy and antibody-based therapeutic cancer products. The Company’s technologies include its investigational Self-Assembly DisAssembly (“SADA”) Pretargeted Radioimmunotherapy Platform (“PRIT”), and bispecific antibodies generated using the Y-BiClone platform. The Company’s broad and advanced product pipeline includes the anti-GD2 therapy DANYELZA® (naxitamab-gqgk), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow after a partial response, minor response, or stable disease to prior therapy. About CD38-SADA PRIT CD38-SADA is a bispecific fusion protein that tightly binds to the CD38 glycoprotein and to 177Lu-tetraxetan (177Lu -DOTA), a “caged” radionuclide. In the first step of pre-targeted radioimmunotherapy, non-radiolabeled CD38-SADA tetramers are infused and bind to CD38-expressing lymphoma cells, and unbound CD38-SADA protein disassembles into low molecular weight monomers that are removed by the kidney. The second infusion delivers the “radioactive payload,” which binds directly to CD38-SADA on tumor cells for localized irradiation. CD38-SADA PRIT with 177Lu-DOTA has demonstrated robust anti-tumor efficacy in preclinical studies and is currently being investigated in adults with relapsed, progressive, or refractory NHL (CD38-expressing B-cell, T-cell, and natural killer cell lymphomas) after at least 2 prior lines of therapy (NCT05994157). Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, statements about our business model, including financial outlook for 2024 and beyond. Words such as ‘‘anticipate,’’ ‘‘believe,’’ “contemplate,” ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ “hope,” ‘‘intend,’’ ‘‘may,’’ ‘‘might,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘should,’’ ‘‘target,’’ “will,” ‘‘would’,’ “guidance,” “goal,” “objective,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company’s business is subject to risks and uncertainties affecting the Company including those described in the “Risk Factors” section included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, and the Company’s Quarterly Report on Form 10-Q for the quarterly periods ended March 31, 2024, and September 30, 2024, and future filings and reports by the Company. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. SADA®, SADA PRIT™, DANYELZA® and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc. Source: Y-mAbs Therapeutics, Inc.